Blockbuster in the balance: Celgene Reblozyl faces FDA panel

In its final days as an independent company, Celgene scored a win last month with the FDA’s approval of rare blood disease med Reblozyl––a potential blockbuster in some analysts’ eyes. Now, the FDA wants a closer look at Reblozyl as a treatment for a group of rare blood cancers.

An FDA advisory committee will review Reblozyl on Dec. 18 to treat patients with myelodysplastic syndromes (MDS), a suite of rare blood cancers in which mutations prevent bone marrow stem cells from making healthy blood cells, leading to chronic anemia.

“Anemia associated with MDS remains a significant area of unmet need for these patients, as current treatment options are limited, consisting primarily of medicines that stimulate the production of erythropoietin and regular RBC transfusions,” new Celgene owner Bristol-Myers Squibb said in a statement.

The FDA set an action date on Reblozyl’s application for April 4. Bristol didn’t disclose what data the FDA was set to review at the advisory committee meeting.

If Reblozly wins an MDS nod, it could reach a $2 billion in yearly sales, according to Jefferies analysts.

In early November, the FDA approved Reblozyl (luspatercept) as an anemia treatment in patients with beta thalassemia who require regular red blood cell transfusions. Though these transfusions are a lifesaving treatment, they can eventually lead to iron overload that can cause organ failure and shorten a patient’s life span.

Reblozyl, co-developed with Cambridge, Massachusetts-based biotech Acceleron, became the first FDA-approved treatment for anemia in beta thalassemia. However, the drug came with label warnings for blood clots, hypertension and embryo-fetal toxicity, the company said.

Serious side effects, including cerebrovascular accidents and deep vein thrombosis, were reported in 1% of trial patients, and one patient reportedly died during clinical testing due to an unconfirmed case of acute myeloid leukemia, Celgene said.

Reblozyl’s win closely followed an FDA approval for Celgene’s JAK inhibitor Inrebic (fedratinib) in August as a first-line or follow-up treatment for myelofibrosis. The once-daily oral drug became the first new treatment in nearly a decade approved to treat the disease, Celgene said.

Inrebic hit the market with a black box warning for serious and fatal encephalopathy, including Wernicke’s. The drugmaker said serious encephalopathy was reported in 1.3% of trial patients treated with Inrebic and one of those patients died.

Both approvals were major wins for Bristol, which is now in the clear on its Celgene merger after the Federal Trade Commission (FTC) voted 3-2 in mid-November to approve the transaction pending the offloading of Celgene psoriasis med Otezla.

Bristol touted Celgene’s “Big 5” late-stage drug candidates––including Reblozly and Inrebic––as major drivers of the merger, with peak sales estimates of more than $5 billion per year. The rest of the list includes multiple sclerosis hopeful ozanimod, which had its application picked up by the FDA in June, and a pair of CAR-T therapies: bluebird bio-partnered bb2121 for multiple myeloma and lisocabtagene maraleucel (liso-cel), formerly called JCAR017, the blood cancer treatment Celgene picked up in its $9 billion Juno Therapeutics buyout.

https://www.fiercepharma.com/pharma/blockbuster-balance-celgene-s-reblozyl-faces-fda-committee-for-mds